- Conditions
- MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
- Interventions
- Aldurazyme (laronidase)
- Drug
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years to 50 Years · Female only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2032
- U.S. locations
- 1
- States / cities
- San Francisco, California
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Showing 25–48
of 24
matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Fabry Disease
- Interventions
- agalsidase alfa
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- Not listed
- U.S. locations
- 25
- States / cities
- Tucson, Arizona • La Jolla, California • Los Angeles, California + 21 more
- Conditions
- Type 1 Gaucher Disease
- Interventions
- Elelyso 60 units/kg
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- Up to 12 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 1
- States / cities
- Fairfax, Virginia
- Conditions
- Fabry Disease
- Interventions
- A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011
- U.S. locations
- 1
- States / cities
- Austin, Texas
- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Interventions
- AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
- Biological · Procedure · Drug + 3 more
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- migalastat HCl
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years to 55 Years · Male only
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2008
- U.S. locations
- 5
- States / cities
- Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
- Conditions
- Fabry Disease
- Interventions
- Enzyme Replacement Therapy (ERT)
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 82 participants
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Gaucher Disease
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- Not listed
- Enrollment
- 200 participants
- Timeline
- 2017 – 2027
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Replagal agalsidase alfa, Replagal
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- 39 Years to 45 Years · Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2003
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease
- Interventions
- Migalastat HCl 20 mg
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 6
- States / cities
- Atlanta, Georgia • Minneapolis, Minnesota • Charlotte, North Carolina + 3 more
- Conditions
- Fabry Disease
- Interventions
- Replagal (agalsidase alfa)
- Biological
- Lead sponsor
- Shire
- Industry
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2013
- U.S. locations
- 5
- States / cities
- Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Day to 100 Years
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2002
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fabry Disease, Proteinuria
- Interventions
- Not listed
- Lead sponsor
- University of Alabama at Birmingham
- Other
- Eligibility
- 14 Years to 95 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2006
- U.S. locations
- 1
- States / cities
- Birmingham, Alabama
- Conditions
- Gaucher's Disease
- Interventions
- CEREDASE™
- Drug
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
- NIH
- Eligibility
- Not listed
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2000
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Niemann-Pick Disease, Type C
- Interventions
- VTS-270
- Drug
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- Up to 6 Months
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Gaucher Disease
- Interventions
- Taliglucerase alfa
- Drug
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 31 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 3
- States / cities
- Coral Springs, Florida • Decatur, Georgia • New York, New York
- Conditions
- Tay-Sachs Disease, Sandhoff Disease
- Interventions
- AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose, AXO-AAV-GM2 High Dose
- Biological
- Lead sponsor
- Terence Flotte
- Other
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2024
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Worcester, Massachusetts
- Conditions
- Fabry Disease
- Interventions
- Not listed
- Lead sponsor
- Baylor Research Institute
- Other
- Eligibility
- 18 Years to 64 Years · Male only
- Enrollment
- 107 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2010 – 2015
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Type I Gaucher Disease
- Interventions
- Ambroxol
- Drug
- Lead sponsor
- Exsar Corporation
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Monmouth Junction, New Jersey
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years and older
- Enrollment
- 85 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2016
- U.S. locations
- 11
- States / cities
- Atlanta, Georgia • Chicago, Illinois • Kansas City, Kansas + 8 more
- Conditions
- Fabry Disease
- Interventions
- migalastat hydrochloride, Placebo
- Drug
- Lead sponsor
- Amicus Therapeutics
- Industry
- Eligibility
- 16 Years to 74 Years
- Enrollment
- 67 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 13
- States / cities
- Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more
- Conditions
- Fabry Disease
- Interventions
- PRX-102 1 mg/kg every two weeks
- Drug
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Industry
- Eligibility
- 2 Years to 17 Years
- Enrollment
- 22 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2031
- U.S. locations
- 6
- States / cities
- Phoenix, Arizona • Atlanta, Georgia • Iowa City, Iowa + 3 more
- Conditions
- Parkinson Disease
- Interventions
- LY3884961, Methylprednisolone, Sirolimus
- Biological · Drug
- Lead sponsor
- Prevail Therapeutics
- Industry
- Eligibility
- 35 Years to 80 Years
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2031
- U.S. locations
- 11
- States / cities
- Scottsdale, Arizona • Murrieta, California • Englewood, Colorado + 7 more